The U.S. Food and Drug Administration (FDA) has recently approved Sanofi’s hemophilia treatment, Qfitlia.
Qfitlia, administered as a subcutaneous injection, is designed for hemophilia patients aged 12 and older. It works by lowering levels of antithrombin, a protein that delays blood clotting, thereby preventing bleeding episodes. The treatment comes with an annual list price of $642,000.
Qfitlia offers a significant advantage over its competitors. While Pfizer’s Hemlibra requires weekly injections and Novo Nordisk’s Alhemo needs daily doses, Qfitlia only needs to be administered bimonthly.
The hemophilia treatment market has been dominated by factor replacement therapy for decades, but recently gene therapies such as CSL’s Hemgenix and BioMarin’s Roctavian have been emerging as promising alternatives..
Hemophilia is a genetic disorder that impairs the body’s ability to produce clotting factors, which are proteins crucial for blood coagulation. This deficiency can lead to spontaneous and severe bleeding, especially following injuries or surgical procedures.
The Centers for Disease Control and Prevention (CDC) reports that approximately 33,000 individuals in the United States are living with hemophilia, with males making up the majority of cases.
Clinical trials for Qfitlia have shown impressive results. The treatment reduced the annual bleeding rate by 90% compared to the control group and demonstrated significant improvements in bleeding events over existing treatments.
